Trial Outcomes & Findings for A Pilot Study of Alemtuzumab (Campath[R]) in Patients With Myelodysplastic Syndrome (NCT NCT00217594)

Last Updated: 2018-10-30

Results Overview

Response to treatment at 3 months after the first dose of alemtuzumab. The parameters for hematologic improvement (HI) and complete response (CR) were defined according to the International Working Group (IWG) criteria. The IWG criteria for HI define specific responses of cytopenias in the 3 hematopoietic lineages: erythroid (HI-E), platelet (HI-P), and neutrophil (HI-N).7 The HIs are measured in patients with pretreatment abnormal values: hemoglobin level less than 110 g/L (11 g/dL) or RBC-transfusion dependence, platelet count less than 100 × 109/L or platelet-transfusion dependence, absolute neutrophil count (ANC) less than 1.0 × 109/L. The parameters for CR include less than 5% marrow blasts without evidence of dysplasia and normalization of peripheral blood counts, including a hemoglobin level of 110 g/L (11 g/dL) or more (in patients not receiving erythropoietin or transfusions), a neutrophil count of 1.5 × 109/L or more, and a platelet count of 100 × 109/L.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

40 participants

Primary outcome timeframe

3 months

Results posted on

2018-10-30

Participant Flow

Out of 40 enrolled subjects, only 31 subjects were evaluable.

Participant milestones

Participant milestones
Measure	Alemtuzumab Patients received a 1-mg test dose of alemtuzumab, and the following day, alemtuzumab was administered at 10 mg/dose intravenously for 10 days
Overall Study STARTED	31
Overall Study COMPLETED	31
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Pilot Study of Alemtuzumab (Campath[R]) in Patients With Myelodysplastic Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Alemtuzumab n=31 Participants Patients received a 1-mg test dose of alemtuzumab, and the following day, alemtuzumab was administered at 10 mg/dose intravenously for 10 days
Age, Continuous	57 years n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants
Race/Ethnicity, Customized Race/Ethnicity · White	24 Participants n=5 Participants
Race/Ethnicity, Customized Race/Ethnicity · African American	2 Participants n=5 Participants
Race/Ethnicity, Customized Race/Ethnicity · Asian	2 Participants n=5 Participants
Race/Ethnicity, Customized Race/Ethnicity · Hispanic	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3 months

Outcome measures

Outcome measures
Measure	Alemtuzumab n=31 Participants Patients received a 1-mg test dose of alemtuzumab, and the following day, alemtuzumab was administered at 10 mg/dose intravenously for 10 days
Response to Treatment - Hematologic Improvement or Complete Response	21 Participants

Adverse Events

Alemtuzumab

Serious events: 15 serious events

Other events: 31 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Alemtuzumab n=31 participants at risk Patients received a 1-mg test dose of alemtuzumab, and the following day, alemtuzumab was administered at 10 mg/dose intravenously for 10 days
Infections and infestations Bacterial pneumonia	3.2% 1/31 • 2 years
Infections and infestations Cellulitis	3.2% 1/31 • 2 years
Infections and infestations Clostridium difficile diarrhea	6.5% 2/31 • 2 years
Infections and infestations Neutropenic fever	9.7% 3/31 • 2 years
Infections and infestations Non-neutropenic fever	6.5% 2/31 • 2 years
Infections and infestations Shingles	3.2% 1/31 • 2 years
Infections and infestations Sinusitis	3.2% 1/31 • 2 years
Infections and infestations URI symptoms	3.2% 1/31 • 2 years
Injury, poisoning and procedural complications Hypotension	3.2% 1/31 • 2 years
Blood and lymphatic system disorders Autoimmune thrombocytopenia	3.2% 1/31 • 2 years
Skin and subcutaneous tissue disorders Molluscum contagiosum skin lesion	3.2% 1/31 • 2 years

Other adverse events

Other adverse events
Measure	Alemtuzumab n=31 participants at risk Patients received a 1-mg test dose of alemtuzumab, and the following day, alemtuzumab was administered at 10 mg/dose intravenously for 10 days
Cardiac disorders Hypertension	3.2% 1/31 • 2 years
General disorders Asthenia	6.5% 2/31 • 2 years
General disorders Fatigue	3.2% 1/31 • 2 years
General disorders Stiffness	3.2% 1/31 • 2 years
Skin and subcutaneous tissue disorders Facial flushing	3.2% 1/31 • 2 years
Skin and subcutaneous tissue disorders Pruritus	3.2% 1/31 • 2 years
Skin and subcutaneous tissue disorders Rash	6.5% 2/31 • 2 years
Skin and subcutaneous tissue disorders Urticaria	9.7% 3/31 • 2 years
Gastrointestinal disorders Diarrhea	3.2% 1/31 • 2 years
Gastrointestinal disorders Nausea	3.2% 1/31 • 2 years
Injury, poisoning and procedural complications Infusion reaction	74.2% 23/31 • 2 years
Infections and infestations Orchitis	3.2% 1/31 • 2 years
Infections and infestations Pilonidal cyst	3.2% 1/31 • 2 years
Infections and infestations Upper respiratory tract	29.0% 9/31 • 2 years
Infections and infestations Mycobacterium chelonae	3.2% 1/31 • 2 years
Blood and lymphatic system disorders Hand swelling	3.2% 1/31 • 2 years
Metabolism and nutrition disorders Decreased phosphate	3.2% 1/31 • 2 years
Metabolism and nutrition disorders Elevated AST, ALT	25.8% 8/31 • 2 years
Metabolism and nutrition disorders Elevated LDH	3.2% 1/31 • 2 years
Metabolism and nutrition disorders Elevated creatinine	3.2% 1/31 • 2 years
Nervous system disorders Dizziness	3.2% 1/31 • 2 years
General disorders Headache	6.5% 2/31 • 2 years
Musculoskeletal and connective tissue disorders Muscle cramps	9.7% 3/31 • 2 years
Nervous system disorders Neuropathic	3.2% 1/31 • 2 years
Renal and urinary disorders Darkened urine	3.2% 1/31 • 2 years

Additional Information

Chris Hourigan MD

NHLBI, NIH

Phone: 301-451-0257

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place